AAV gene therapy for hereditary spastic paraplegia type 50: a phase 1 trial in a single patient
AAV基因疗法治疗50型遗传性痉挛性截瘫：一项单例患者的I期临床试验
期刊:Nature Medicine
影响因子:50
doi:10.1038/s41591-024-03078-4
Dowling, James J; Pirovolakis, Terry; Devakandan, Keshini; Stosic, Ana; Pidsadny, Mia; Nigro, Elisa; Sahin, Mustafa; Ebrahimi-Fakhari, Darius; Messahel, Souad; Varadarajan, Ganapathy; Greenberg, Benjamin M; Chen, Xin; Minassian, Berge A; Cohn, Ronald; Bonnemann, Carsten G; Gray, Steven J
